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Posted On: 04/01/2020 9:30:20 AM
Post# of 153973
Why 342 patients
In clinical trials, there is a concept of power. There is a statistical calculation of the number of patients that will be needed to produce an efficacy signal. Contributing factors are the expected variability of outcomes.
While the press release did not say it, Nader mentioned in the proactive video yesterday that this would be a phase 3 file which is one that IF successful would lead to approval upon data submission.
Phase 3 files typically have the highest number of patients.
The good news is the endpoint is short in two weeks but data collection, coalition and submission will take time.
There is also normally am interim data review that a small group gets to see unblinded data to ensure safety and efficacy part way through the trial.
This will be an important activity.
Of course the data on the first 10 in the compassionate use will be an early indication although not considered a gold standard double blind placebo controlled study.
All. IMO
In clinical trials, there is a concept of power. There is a statistical calculation of the number of patients that will be needed to produce an efficacy signal. Contributing factors are the expected variability of outcomes.
While the press release did not say it, Nader mentioned in the proactive video yesterday that this would be a phase 3 file which is one that IF successful would lead to approval upon data submission.
Phase 3 files typically have the highest number of patients.
The good news is the endpoint is short in two weeks but data collection, coalition and submission will take time.
There is also normally am interim data review that a small group gets to see unblinded data to ensure safety and efficacy part way through the trial.
This will be an important activity.
Of course the data on the first 10 in the compassionate use will be an early indication although not considered a gold standard double blind placebo controlled study.
All. IMO
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